{"title":"Safety and efficacy of pemigatinib in patients with cholangiocarcinoma: a systematic review.","authors":"Warda Rasool, Yassine Alami Idrissi, Owais Ahmad, Shree Rath, Fatima Saeed, Mohamed Saad Sayed, Bhavya Sharma, Arwa Amer Ibrahim, Ajanta Kumari, Irfan Ullah, Anwaar Saeed","doi":"10.21037/jgo-2024-923","DOIUrl":null,"url":null,"abstract":"<p><strong>Background: </strong>Cholangiocarcinoma (CCA) is an aggressive bile duct cancer with limited therapeutic options and poor prognosis. pemigatinib, a selective <i>FGFR</i> inhibitor, has emerged as a promising targeted therapy for CCA patients harboring <i>FGFR2</i> fusions or rearrangements. This systematic review evaluated the safety and efficacy of pemigatinib in this patient population.</p><p><strong>Methods: </strong>A comprehensive systematic review was conducted across PubMed, Scopus, Embase, Cochrane Library, and Web of Science to identify studies investigating pemigatinib in CCA patients. Five studies involving a total of 459 patients met the inclusion criteria.</p><p><strong>Results: </strong>Pemigatinib demonstrated an overall objective response rate (ORR) of 43.2%, with a complete response (CR) achieved in 3% of patients. Stable disease was observed in 36.9% of patients, while 14.9% experienced disease progression. Median progression-free survival (PFS) varied across studies, due to differences in patient cohorts. The most common adverse effects (AEs) included hyperphosphatemia (48%), diarrhea (28.6%), fatigue (33%), and dry eyes (20.1%).</p><p><strong>Conclusions: </strong>This systematic review suggests that pemigatinib has modest therapeutic efficacy in CCA patients, with a considerable proportion achieving disease control. However, the ORR of less than 50% highlights the potential need for combination or sequential therapies to improve outcomes. Close monitoring and management of AEs, particularly hyperphosphatemia, are crucial for optimizing treatment. Further large-scale randomized trials and research are warranted to identify predictive biomarkers and optimize pemigatinib-based treatment strategies for CCA patients with FGFR2 alterations.</p>","PeriodicalId":15841,"journal":{"name":"Journal of gastrointestinal oncology","volume":"16 2","pages":"699-710"},"PeriodicalIF":2.0000,"publicationDate":"2025-04-30","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12078806/pdf/","citationCount":"0","resultStr":null,"platform":"Semanticscholar","paperid":null,"PeriodicalName":"Journal of gastrointestinal oncology","FirstCategoryId":"3","ListUrlMain":"https://doi.org/10.21037/jgo-2024-923","RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"2025/4/25 0:00:00","PubModel":"Epub","JCR":"Q3","JCRName":"GASTROENTEROLOGY & HEPATOLOGY","Score":null,"Total":0}
引用次数: 0
Abstract
Background: Cholangiocarcinoma (CCA) is an aggressive bile duct cancer with limited therapeutic options and poor prognosis. pemigatinib, a selective FGFR inhibitor, has emerged as a promising targeted therapy for CCA patients harboring FGFR2 fusions or rearrangements. This systematic review evaluated the safety and efficacy of pemigatinib in this patient population.
Methods: A comprehensive systematic review was conducted across PubMed, Scopus, Embase, Cochrane Library, and Web of Science to identify studies investigating pemigatinib in CCA patients. Five studies involving a total of 459 patients met the inclusion criteria.
Results: Pemigatinib demonstrated an overall objective response rate (ORR) of 43.2%, with a complete response (CR) achieved in 3% of patients. Stable disease was observed in 36.9% of patients, while 14.9% experienced disease progression. Median progression-free survival (PFS) varied across studies, due to differences in patient cohorts. The most common adverse effects (AEs) included hyperphosphatemia (48%), diarrhea (28.6%), fatigue (33%), and dry eyes (20.1%).
Conclusions: This systematic review suggests that pemigatinib has modest therapeutic efficacy in CCA patients, with a considerable proportion achieving disease control. However, the ORR of less than 50% highlights the potential need for combination or sequential therapies to improve outcomes. Close monitoring and management of AEs, particularly hyperphosphatemia, are crucial for optimizing treatment. Further large-scale randomized trials and research are warranted to identify predictive biomarkers and optimize pemigatinib-based treatment strategies for CCA patients with FGFR2 alterations.
背景：胆管癌（CCA）是一种侵袭性胆管癌，治疗方案有限，预后差。pemigatinib是一种选择性FGFR抑制剂，已成为具有FGFR2融合或重排的CCA患者的一种有希望的靶向治疗方法。本系统综述评估了培伽替尼在该患者群体中的安全性和有效性。方法：对PubMed、Scopus、Embase、Cochrane Library和Web of Science进行全面的系统评价，以确定调查pemigatinib在CCA患者中的应用的研究。5项共涉及459例患者的研究符合纳入标准。结果：Pemigatinib的总体客观缓解率（ORR）为43.2%，完全缓解（CR）在3%的患者中实现。36.9%的患者病情稳定，14.9%的患者病情进展。由于患者队列的差异，各研究的中位无进展生存期（PFS）有所不同。最常见的不良反应（ae）包括高磷血症（48%）、腹泻（28.6%）、疲劳（33%）和眼干（20.1%）。结论：本系统综述提示，培伽替尼对CCA患者有中等疗效，有相当比例的患者达到疾病控制。然而，低于50%的ORR表明，可能需要联合或顺序治疗来改善结果。密切监测和管理不良事件，特别是高磷血症，对优化治疗至关重要。对于FGFR2改变的CCA患者，需要进一步的大规模随机试验和研究来确定预测性生物标志物，并优化基于培伽替尼的治疗策略。
期刊介绍：
ournal of Gastrointestinal Oncology (Print ISSN 2078-6891; Online ISSN 2219-679X; J Gastrointest Oncol; JGO), the official journal of Society for Gastrointestinal Oncology (SGO), is an open-access, international peer-reviewed journal. It is published quarterly (Sep. 2010- Dec. 2013), bimonthly (Feb. 2014 -) and openly distributed worldwide.
JGO publishes manuscripts that focus on updated and practical information about diagnosis, prevention and clinical investigations of gastrointestinal cancer treatment. Specific areas of interest include, but not limited to, multimodality therapy, markers, imaging and tumor biology.
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